Revolutionary therapy: Researchers give muscles back!

Revolutionary therapy: Researchers give muscles back!

In a significant breakthrough in combating muscle loss, two Berlin researchers, professor Simone Spuler and Dr. Helena Escobar Fernandez, made promising progress. Currently over 1100 people in Berlin are affected by muscular dystrophy, an illness that leads to progressive loss of muscle strength. Initially, the disease is creeping, but many affected people lose the ability to move normally at a young age and are dependent on a wheelchair at the age of 40.

The decisive factor in therapy is the protein dysferlin, which does not work properly in the patient and is responsible for repairing cell membranes. With the innovative CrisPR-CAS9 process, which was awarded the Nobel Prize in 2020, the scientists managed to repair the defective gene in the laboratory and thus stimulate the growth of the tissue. Two patients could be successfully treated with genetically corrected muscle stem cells. This method could soon also be used in humans, as the researchers explained in their report because the first clinical studies could start in 2025.

First successes and future studies

The experiments not only delivered promising results in the laboratory: With the help of the CrisPR-CAS9 process, muscle stem cells could also be successfully removed, genetically processed and transplanted. The positive development arouses hopes that there could soon be therapies that not only restore the function of the muscles, but also stop the progression of the disease. This would be a revolutionary progress for many affected people who suffer from the effects of muscular dystrophy, said Spuler. However, research is still in the early phases, and the complexity of the human body requires careful planning and implementation of the first clinical studies to ensure the safety and effectiveness of the new treatment methods. ”We are very modest with one or two muscles, but if this therapy works, it could help heal these muscles,” emphasizes Spuler, the ambitious approach of her team, which continues to focus on the development of effective therapies.

These results and the upcoming clinical studies not only show the impressive progress in gene research, but also confirm the importance of experts such as Spuler and Fernandez, who work tirelessly on solutions for this devastating disease. For further details about the team and its projects, interested readers can view the information on the website of the Max Delbrück Center, which offers comprehensive insights into this exciting research.

Further information can be found on ​​BZ Berlin and on the official page of Max-delbrück-centrums .

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